Work

Decision Analysis of Treatment Strategies in Children With Severe Sickle Cell Disease

Year: 2009

Type: article

Abstract: We designed a decision analysis model comparing 4 treatment strategies for severe sickle cell disease: no intervention, hydroxyurea (HU), chronic transfusion, or stem cell transplant (SCT). The treatment strategy associated with the highest average utility (quality of life) was SCT (0.85). Average utilities for no treatment, chronic transfusion, and HU were 0.68, 0.71, and 0.80, respectively. Our model was quite sensitive to quality-of-life estimates, indicating that a true comparison of HU and transplantation cannot occur until investigators directly measure the health-related quality of life in children with sickle cell disease during HU therapy and after SCT.

Source: Journal of Pediatric Hematology/Oncology

DOI: 10.1097/mph.0b013e3181b83cab

Authors: Sarah H. O'Brien, Jane S. Hankins

Institutions: Nationwide Children's Hospital, Abigail Wexner Research Institute, St. Jude Children's Research Hospital

Language: English

Topic: Hemoglobinopathies and Related Disorders

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

Funders: National Heart, Lung, and Blood Institute, National Institute of Diabetes and Digestive and Kidney Diseases